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Therapy: Gene Therapy
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Total 5568 results found since Jan 2013.
RNA Interference and Neuromuscular Diseases: A Focus on Hereditary Transthyretin Amyloidosis
Curr Gene Ther. 2023 Sep 13. doi: 10.2174/1566523223666230913110011. Online ahead of print.ABSTRACTNeuromuscular diseases are severe disorders affecting the peripheral nervous system, usually driving to death in a limited time. Many new drugs, through RNA-interference technology, are revolutionizing the prognosis and quality of life for these patients. Nevertheless, given the increased life expectancy, some new issues and phenotypes are expected to be revealed. In the transthyretin-mediated hereditary amyloidosis (ATTR-v, "v" for "variant"), the RNA interference was demonstrated to effectively reduce the hepatic synthesis ...
Source: Current Gene Therapy - September 15, 2023 Category: Genetics & Stem Cells Authors: Marco Ceccanti Maurizio Inghilleri Source Type: research
Prospects and challenges of CRISPR/Cas9 gene-editing technology in cancer research
Clin Genet. 2023 Sep 14. doi: 10.1111/cge.14424. Online ahead of print.ABSTRACTCancer, one of the leading causes of death, usually commences and progresses as a result of a series of gene mutations and dysregulation of expression. With the development of clustered regularly interspaced palindromic repeat (CRISPR)/Cas9 gene-editing technology, it is possible to edit and then decode the functions of cancer-related gene mutations, markedly advance the research of biological mechanisms and treatment of cancer. This review summarizes the mechanism and development of CRISPR/Cas9 gene-editing technology in recent years and descri...
Source: Clinical Genetics - September 14, 2023 Category: Genetics & Stem Cells Authors: Li Ning Jiahui Xi Yin Zi Min Chen Qingjian Zou Xiaoqing Zhou Chengcheng Tang Source Type: research
Breast Cancer Organoids Derived from Patients: A Platform for Tailored Drug Screening
Biochem Pharmacol. 2023 Sep 12:115803. doi: 10.1016/j.bcp.2023.115803. Online ahead of print.ABSTRACTBreast cancer stands as the most prevalent and heterogeneous malignancy affecting women globally, posing a substantial health concern. Enhanced comprehension of tumor pathology and the development of novel therapeutics are pivotal for advancing breast cancer treatment. Contemporary breast cancer investigation heavily leans on in vivo models and conventional cell culture techniques. Nonetheless, these approaches often encounter high failure rates in clinical trials due to species disparities and tissue structure variations. ...
Source: Biochemical Pharmacology - September 14, 2023 Category: Drugs & Pharmacology Authors: Yen-Dun Tony Tzeng Jui-Hu Hsiao Ling-Ming Tseng Ming-Feng Hou Chia-Jung Li Source Type: research
Prospects and challenges of CRISPR/Cas9 gene-editing technology in cancer research
Clin Genet. 2023 Sep 14. doi: 10.1111/cge.14424. Online ahead of print.ABSTRACTCancer, one of the leading causes of death, usually commences and progresses as a result of a series of gene mutations and dysregulation of expression. With the development of clustered regularly interspaced palindromic repeat (CRISPR)/Cas9 gene-editing technology, it is possible to edit and then decode the functions of cancer-related gene mutations, markedly advance the research of biological mechanisms and treatment of cancer. This review summarizes the mechanism and development of CRISPR/Cas9 gene-editing technology in recent years and descri...
Source: Clinical Genetics - September 14, 2023 Category: Genetics & Stem Cells Authors: Li Ning Jiahui Xi Yin Zi Min Chen Qingjian Zou Xiaoqing Zhou Chengcheng Tang Source Type: research
Breast Cancer Organoids Derived from Patients: A Platform for Tailored Drug Screening
Biochem Pharmacol. 2023 Sep 12:115803. doi: 10.1016/j.bcp.2023.115803. Online ahead of print.ABSTRACTBreast cancer stands as the most prevalent and heterogeneous malignancy affecting women globally, posing a substantial health concern. Enhanced comprehension of tumor pathology and the development of novel therapeutics are pivotal for advancing breast cancer treatment. Contemporary breast cancer investigation heavily leans on in vivo models and conventional cell culture techniques. Nonetheless, these approaches often encounter high failure rates in clinical trials due to species disparities and tissue structure variations. ...
Source: Biochemical Pharmacology - September 14, 2023 Category: Drugs & Pharmacology Authors: Yen-Dun Tony Tzeng Jui-Hu Hsiao Ling-Ming Tseng Ming-Feng Hou Chia-Jung Li Source Type: research
Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products
Curr Gene Ther. 2023 Sep 11. doi: 10.2174/1566523223666230911120922. Online ahead of print.ABSTRACTThe success of gene therapy attempts is controversial and inconclusive. Currently, it is popular among the public, the scientific community, and manufacturers of Gene Therapy Medical Products. In the absence of any remedy or treatment options available for untreatable inborn metabolic orphan or genetic diseases, cancer, or brain diseases, gene therapy treatment by genoceuticals and T-cells for gene editing and recovery remains the preferred choice as the last hope. A new concept of "Genoceutical Gene Therapy" by using orphan ...
Source: Current Gene Therapy - September 13, 2023 Category: Genetics & Stem Cells Authors: Rakesh Sharma Source Type: research
Translational strategies and systems biology insights for blood-brain barrier opening and delivery in brain tumors and Alzheimer's disease
Biomed Pharmacother. 2023 Sep 11;167:115450. doi: 10.1016/j.biopha.2023.115450. Online ahead of print.ABSTRACTThe blood-brain barrier (BBB) plays a critical role in determining the effectiveness of systemic treatments for brain diseases. Over the years, several innovative approaches in BBB opening and drug delivery have been developed and progressed into clinical testing phases, including focused ultrasound (FUS) with circulating microbubbles, mannitol-facilitated delivery of anti-neoplastic drugs, receptor-mediated transcytosis (RMT) by antibody-drug conjugates (ADCs), and viral vectors for gene therapy. We provided a com...
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - September 13, 2023 Category: Drugs & Pharmacology Authors: Gefei Song Pierce Plumlee Ju Young Ahn Stephen Tc Wong Hong Zhao Source Type: research
Applications of tissue-specific and cancer-selective gene promoters for cancer diagnosis and therapy
Adv Cancer Res. 2023;160:253-315. doi: 10.1016/bs.acr.2023.03.005. Epub 2023 Apr 20.ABSTRACTCurrent treatment of solid tumors with standard of care chemotherapies, radiation therapy and/or immunotherapies are often limited by severe adverse toxic effects, resulting in a narrow therapeutic index. Cancer gene therapy represents a targeted approach that in principle could significantly reduce undesirable side effects in normal tissues while significantly inhibiting tumor growth and progression. To be effective, this strategy requires a clear understanding of the molecular biology of cancer development and evolution and develo...
Source: Advances in Cancer Research - September 13, 2023 Category: Cancer & Oncology Authors: Amit Kumar Swadesh K Das Luni Emdad Paul B Fisher Source Type: research
Gene-environment interactions that influence CVD, lipid traits, obesity, diabetes, and hypertension appear to be able to influence gene therapy
Mol Aspects Med. 2023 Sep 11;94:101213. doi: 10.1016/j.mam.2023.101213. Online ahead of print.ABSTRACTMost mind boggling diseases are accepted to be impacted by both genetic and environmental elements. As of late, there has been a flood in the improvement of different methodologies, concentrate on plans, and measurable and logical techniques to examine gene-environment cooperations (G × Es) in enormous scope studies including human populaces. The many-sided exchange between genetic elements and environmental openings has long charmed the consideration of clinicians and researchers looking to grasp the complicated starting...
Source: Molecular Medicine - September 13, 2023 Category: Molecular Biology Authors: Moataz Dowaidar Source Type: research
Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products
Curr Gene Ther. 2023 Sep 11. doi: 10.2174/1566523223666230911120922. Online ahead of print.ABSTRACTThe success of gene therapy attempts is controversial and inconclusive. Currently, it is popular among the public, the scientific community, and manufacturers of Gene Therapy Medical Products. In the absence of any remedy or treatment options available for untreatable inborn metabolic orphan or genetic diseases, cancer, or brain diseases, gene therapy treatment by genoceuticals and T-cells for gene editing and recovery remains the preferred choice as the last hope. A new concept of "Genoceutical Gene Therapy" by using orphan ...
Source: Current Gene Therapy - September 13, 2023 Category: Genetics & Stem Cells Authors: Rakesh Sharma Source Type: research
Translational strategies and systems biology insights for blood-brain barrier opening and delivery in brain tumors and Alzheimer's disease
Biomed Pharmacother. 2023 Sep 11;167:115450. doi: 10.1016/j.biopha.2023.115450. Online ahead of print.ABSTRACTThe blood-brain barrier (BBB) plays a critical role in determining the effectiveness of systemic treatments for brain diseases. Over the years, several innovative approaches in BBB opening and drug delivery have been developed and progressed into clinical testing phases, including focused ultrasound (FUS) with circulating microbubbles, mannitol-facilitated delivery of anti-neoplastic drugs, receptor-mediated transcytosis (RMT) by antibody-drug conjugates (ADCs), and viral vectors for gene therapy. We provided a com...
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - September 13, 2023 Category: Drugs & Pharmacology Authors: Gefei Song Pierce Plumlee Ju Young Ahn Stephen Tc Wong Hong Zhao Source Type: research
Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products
Curr Gene Ther. 2023 Sep 11. doi: 10.2174/1566523223666230911120922. Online ahead of print.ABSTRACTThe success of gene therapy attempts is controversial and inconclusive. Currently, it is popular among the public, the scientific community, and manufacturers of Gene Therapy Medical Products. In the absence of any remedy or treatment options available for untreatable inborn metabolic orphan or genetic diseases, cancer, or brain diseases, gene therapy treatment by genoceuticals and T-cells for gene editing and recovery remains the preferred choice as the last hope. A new concept of "Genoceutical Gene Therapy" by using orphan ...
Source: Current Gene Therapy - September 13, 2023 Category: Genetics & Stem Cells Authors: Rakesh Sharma Source Type: research
Applications of tissue-specific and cancer-selective gene promoters for cancer diagnosis and therapy
Adv Cancer Res. 2023;160:253-315. doi: 10.1016/bs.acr.2023.03.005. Epub 2023 Apr 20.ABSTRACTCurrent treatment of solid tumors with standard of care chemotherapies, radiation therapy and/or immunotherapies are often limited by severe adverse toxic effects, resulting in a narrow therapeutic index. Cancer gene therapy represents a targeted approach that in principle could significantly reduce undesirable side effects in normal tissues while significantly inhibiting tumor growth and progression. To be effective, this strategy requires a clear understanding of the molecular biology of cancer development and evolution and develo...
Source: Advances in Cancer Research - September 13, 2023 Category: Cancer & Oncology Authors: Amit Kumar Swadesh K Das Luni Emdad Paul B Fisher Source Type: research
